Press Releases

Label Expansion Decisions Expected from U.S. and European Regulatory Authorities in Q4 SAN RAFAEL, Calif., Sept. 21, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global...

European Commission Approval Decision Expected Q4 2023 Opinion Based on Positive Results from Global Phase 2 and Ongoing Extension Study U.S. Food and Drug Administration PDUFA Target Action Date...

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, will update the investor community on the company's research...

BioMarin Leadership and External Medical Experts to Discuss 7 Product Candidates in Clinical Development as well as ROCTAVIAN and VOXZOGO New Indication Opportunities R&D Day to be Webcast...

Citi's 18th Annual BioPharma Conference on September 6 at 3:30pm ET Morgan Stanley 21st Annual Global Healthcare Conference on September 11 at 10:00am ET Baird's 2023 Global Healthcare Conference...

First Person Treated in Germany; Reimbursement Discussions on Track in France and Italy In the U.S., ROCTAVIAN Now Commercially Available and Hemophilia Treatment Centers Have Begun Screening...

VOXZOGO® Growth Continued in the Second Quarter Driven by Global Demand Resulting in Increased Full Year 2023 Guidance Pivotal Program with VOXZOGO in New, Potential Second Indication,...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Monday, July...

ROCTAVIAN is a One-Time, Single-Dose Treatment Available for Adults with Severe Hemophilia A to Control Bleeds ROCTAVIAN's Approval was Based on Durability, Efficacy and Safety Results from the...

New Data to be Presented for ROCTAVIAN from Phase 3 GENEr8-1 Study, Including Data on the Impact of ROCTAVIAN on Quality of Life and Musculoskeletal Health for Adults with Severe Hemophilia A SAN...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Brian Mueller, Executive Vice President, Chief Financial Officer of BioMarin will present at the Bank of America 2023 Health Care...

Growth of VOXZOGO® Accelerated with Rapid Uptake in Japan and Brazil Resulting in Increased Full-year 2023 Guidance Full-year 2023 Top-line and Bottom-line Financial Guidance Reaffirmed;...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday,...

New Analyses from Phase 2 and Phase 3 Extension Studies Continue to Demonstrate Sustained Improvement in Growth with no Change in Safety Profile SAN RAFAEL, Calif., March 14, 2023 /PRNewswire/ --...

The Supplemental New Drug Application is Based on Positive Results from Global Randomized Phase 2 Study in Infants and Young Children FDA set PDUFA Target Action Date of October 21, 2023 SAN...

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that it received notice this afternoon from...

Cowen Healthcare Conference on March 8 in Boston Barclays Global Healthcare Conference on March 15 in Miami Jefferies Biotech on the Bay Summit on March 16 in Miami SAN RAFAEL, Calif., March 2, 2023 /

VOXZOGO $169 Million Contribution Drives Record Full Year 2022 Total Revenues of $2.1 Billion In 2023, More than 15% Growth in Total Revenues and Approximately 30% Growth in Net Income Expected...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer and Brian Mueller, Executive Vice President, Chief Financial Officer...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Monday,...

- Mean Annualized Bleed Rate Reduced by 80% from Baseline and Factor VIII Usage Reduced by 94% in Year 3 Compared to Baseline - 92% of Patients off Prophylaxis at the End of Year 3 - First...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer, will present at the 41st Annual J.P. Morgan Healthcare Conference on...

BioMarin Submits Supplemental New Drug Application to U.S. Food and Drug Administration to Expand Label to Treat Children with Achondroplasia Under the Age of 5 Submissions Based on Favorable...

FDA No Longer Plans to Hold an Advisory Committee Meeting, as Previously Planned, to Discuss the Biologics License Application (BLA) BioMarin Remains on Track to Host Scheduled Manufacturing...

FDA Pre-Licensure Inspection of Gene Therapy Manufacturing Facility Scheduled FDA Requests Submission of Upcoming 3-Year Data Analysis from Phase 3 GENEr8-1 Study Current PDUFA Target Action Date...

Credit Suisse 31st Annual Healthcare Conference 2022: November 8th, 8:35amPT/11:35amET, in Rancho Palos Verdes, CA 13th Annual Jefferies Global Healthcare Conference 2022: November 16th, 8:35amGMT, in

VOXZOGO® $48 Million Contribution in the Quarter Results in BioMarin Raising Full-year 2022 VOXZOGO Net Product Revenue Guidance to Between $140 Million and $170 Million U.S. Biologics...

If Approved, Would Be 1st Gene Therapy in U.S. for Treatment of Severe Hemophilia A PDUFA Target Action Date is March 31, 2023 SAN RAFAEL, Calif., Oct. 12, 2022 /PRNewswire/ -- BioMarin...

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced its decision to redesign the organization to better focus investments that advance its R&D pipeline, maximize recent commercial launch...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday,...

BLA Includes Substantial Body of Data from Pivotal Phase 3 and Ongoing Phase 1/2 Studies If Approved, Would Be 1st Gene Therapy in U.S. for Treatment of Severe Hemophilia A SAN RAFAEL, Calif.,...

Institute for Clinical and Economic Review (ICER) Base-Case Model Report Results in $4 Million Savings Versus Emicizumab Prophylaxis Per Patient Over a Lifetime Biologics License Application (BLA)...

Maintains Orphan Drug Designation (ODD) in the EU Providing 10-years of Market Exclusivity Significant Benefit Over Existing Therapies for Patients with Severe Hemophilia A in EU Based on EMA...

VOXZOGO™ $34 Million Contribution Drives Total Revenues of $534 Million in the Quarter; Total Revenues Grew 13% Year-Over-Year, Excluding Kuvan BioMarin Expects that ROCTAVIAN™ (valoctocogene...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday,...

Commitment to Advancing Care for People with Hemophilia A Demonstrated with Largest and Longest, Ongoing Clinical Development Program for any Gene Therapy in Hemophilia A European Commission...

European Commission Approval Expected Q3 2022 1st Gene Therapy for Treatment of Hemophilia A Recommended for Approval in Europe More than 20,000 Adults with Severe Hemophilia A in BioMarin...

No Lower Age Restriction for Treatment Japan Accounts for Approximately Half of the 1,500 Patient Opportunity in APAC Region SAN RAFAEL, Calif., June 21, 2022 /PRNewswire/ -- BioMarin...

Improvement in Height Z-Score and Annualized Growth Velocity Observed, Consistent with Children Over 5 Interactions with Health Authorities Planned for H2 2022 to Discuss Expanding Access to...

95% Reduction in Mean Annualized Bleed Rate (ABR) and 96% Reduction in Mean Annualized Factor VIII Usage from Baseline Through Year 6 in 6e13 vg/kg Dose Cohort 91% Reduction in Mean ABR and 93%...

Humaira Serajuddin Named to Newly Created Role of Senior Vice President, Chief Marketing Officer Erin Burkhart, Named Group Vice President, Chief Accounting Officer SAN RAFAEL, Calif., May 5, 2022...

Ongoing Clinical Development Program Represents Largest and Longest Development Program for any Gene Therapy in Hemophilia A, Demonstrates Commitment to Advancing Care for People with Hemophilia A...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer, will present at the BofA Securities Healthcare Conference on May 12...

BioMarin Reports Record First Quarter Total Revenues of $519 Million Driven by a $20 Million VOXZOGO Contribution; Total Revenues Grew 11% Year-over-Year, Excluding Kuvan Full-year 2022 Financial...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday,...

New England Journal of Medicine Publishes Editorial: Prepare the Way for Hemophilia A Gene Therapy Significantly Reduced Bleeding and Factor VIII Utilization Compared to Baseline Factor VIII...

Financial Highlights (in millions of U.S. dollars, except per share data, unaudited) Three Months Ended December 31, Twelve Months Ended December 31, 2021 2020 % Change 2021 2020 % Change Total...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced updates on its investigational gene therapy programs in clinical development. In February 2022, the Company received additional requests from...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in two upcoming virtual conferences. An audio webcast of the presentations will be available live. You...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday,...

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that it has entered into a definitive agreement with an undisclosed purchaser to sell the Rare Pediatric Disease Priority Review Voucher...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the Company presented positive results from a two-year analysis of the Phase 3 GENEr8-1 study and an overall safety update of...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the Company will be presenting data in several presentations on valoctocogene roxaparvovec, an investigational gene therapy for the...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer, will present at two virtual conferences in January. To access the...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) and Skyline Therapeutics (formerly Geneception), a gene and cell therapy company focused on developing novel treatments for unmet medical needs, today...

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN), a global leader in providing therapies for rare genetic diseases, today announced the appointment of former CEO of Celgene Corporation, Mark Alles, to...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will host a virtual R&D Day beginning at 8:00am PT/11:00am ET today, Tuesday, November 30, 2021. BioMarin management and external experts will provide...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in two upcoming virtual investor conferences. An audio webcast of the presentations will be available...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval to VOXZOGO™ (vosoritide) for Injection, indicated to...

Financial Highlights (in millions of U.S. dollars, except per share data, unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 % Change 2021 2020 % Change Total...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in four upcoming virtual investor conferences. An audio webcast of the presentations will be available...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday,...

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the Company hired two industry veterans Harold S. Bernstein, M.D., Ph.D. as Senior Vice President, Chief Medical Officer and Head of...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the U.S Food and Drug Administration (FDA) placed a clinical hold on the BMN 307 Phearless Phase 1/2 study. The Phearless study is...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Commission (EC) has granted marketing authorization for VOXZOGO® (vosoritide), a once daily injection to treat...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in three upcoming virtual investor conferences. An audio webcast of the presentations will be available...

Financial Highlights (in millions of U.S. dollars, except per share data, unaudited) Three Months Ended June 30, Six Months Ended June 30, 2021 2020 % Change 2021 2020 % Change Total Revenues $ 501.7

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today new data for valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A, from its...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced new data for valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, in its...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for its investigational gene...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday,...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced three oral presentations and nine poster presentations related to valoctocogene roxaparvovec, an investigational gene therapy for the...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its investigational...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization for...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in the BofA Securities 2021 Napa Biopharma Conference on June 16, 2021 at 3:30pm ET. An audio webcast...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in two upcoming virtual conferences. An audio webcast of the presentations will be available live. You...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) has granted its request for accelerated assessment of valoctocogene roxaparvovec, for adults...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today an update to its previously reported results from an open-label Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in two upcoming virtual conferences. An audio webcast of the presentations will be available live. You...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced three oral and three poster presentations on valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in two upcoming virtual conferences. An audio webcast of the presentations will be available live. You...

Financial Highlights (in millions of U.S. dollars, except per share data, unaudited) Three Months Ended March 31, 2021 2020 % Change Total Revenues $ 486.0 $ 502.1 (3) % Net Product Revenues...

The Allen Institute and BioMarin Pharmaceutical Inc. announced a collaboration today that will use technologies developed at the Allen Institute to create new gene therapies aimed at rare genetic...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) presented new and updated data at the 2021 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting that demonstrates...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Thursday,...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that data from the open-label long-term extension of the Phase 3 study of 15 µg/kg dose of vosoritide was presented at an oral...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene...

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the company has completed full enrollment in a global Phase 2 randomized, placebo-controlled study of vosoritide, an...

Financial Highlights (in millions of U.S. dollars, except per share data, unaudited) Three Months Ended December 31, Twelve Months Ended December 31, 2020 2019 % Change 2020 2019 % Change Total...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in four upcoming virtual conferences. An audio webcast of the presentations will be available live. You...

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN), a global leader in providing therapies for rare genetic diseases, today announced the appointment of former Goldman Sachs partner and executive in the...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced the Company received the top score of 100 on the Human Rights Campaign Foundation's 2021 Corporate Equality Index (CEI), the nation's...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Thursday,...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in two upcoming virtual conferences. An audio webcast of the presentations will be available live. You...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that children in the open-label long-term extension of the Phase 3 study of vosoritide, an investigational, once daily injection analog...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in two upcoming virtual conferences. An audio webcast of the presentations will be available live. You...

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) and Deep Genomics today announced that the companies have entered into a preclinical collaboration that will use Deep Genomics' artificial intelligence...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in three upcoming virtual conferences. An audio webcast of the presentations will be available live....